In short
DIAG723 is an investigational medicine described as a human bispecific monoclonal antibody targeting ALK1 and BMPRII. It is being studied in adults with hereditary hemorrhagic telangiectasia (HHT), a rare condition in which abnormal blood vessels form. One clinical trial, a Phase 1/2 study with 93 planned participants, is currently authorised to evaluate the safety and tolerability of DIAG723.
Key points
- DIAG723 is an investigational bispecific monoclonal antibody targeting ALK1 and BMPRII.
- The only registered trial is a Phase 1/2 study in adults with hereditary hemorrhagic telangiectasia (HHT).
- The trial plans to enrol 93 participants and is currently authorised.
- DIAG723 is given by subcutaneous injection; the study also uses a sodium chloride solution for injection.
- The main goal is to measure safety and tolerability, including treatment-emergent adverse events and dose-limiting toxicities.
What is DIAG723?
DIAG723 is an investigational medicine. The full name given in the trial record is human bispecific monoclonal antibody targeting ALK1 and BMPRII. A bispecific monoclonal antibody is a laboratory-made protein designed to attach to two different targets at the same time. In this case, the two targets are ALK1 and BMPRII, which are proteins involved in blood vessel formation. DIAG723 is not an authorised medicine; it is being studied in clinical research.
How does DIAG723 work?
The trial record describes DIAG723 as a bispecific monoclonal antibody targeting ALK1 and BMPRII. These two proteins are part of a signalling pathway that helps control how blood vessels grow and develop. In hereditary hemorrhagic telangiectasia, this pathway does not work correctly, which leads to abnormal connections between arteries and veins. By binding to both ALK1 and BMPRII, DIAG723 is designed to influence this pathway. The exact effects in people are still being investigated in the current trial.
Which conditions is DIAG723 studied for?
- Hereditary hemorrhagic telangiectasia (HHT) — a rare inherited condition in which blood vessels do not develop normally, leading to fragile connections between arteries and veins that can bleed.
This is the only condition listed in the registered trial for DIAG723.
How is DIAG723 given?
In the registered trial, DIAG723 is given as a subcutaneous injection, which means it is injected under the skin. The trial also lists a sodium chloride solution for injection, which is a salt solution commonly used as a placebo or for dilution. The trial is designed to test single ascending doses in one part and multiple-dose regimens in other parts.
What do the clinical trials show?
One clinical trial of DIAG723 is registered. It is a Phase 1/2 study in adults with hereditary hemorrhagic telangiectasia (HHT). The trial is currently authorised, meaning it has received approval to proceed but results are not yet available. It plans to enrol 93 participants.
The study is described as interventional. Participants receive DIAG723 by subcutaneous injection. The design includes single ascending doses (Part A) and multiple-dose regimens (Parts B and C). This means early participants receive one dose, and later parts test repeated dosing.
What are the trials measuring?
The primary outcomes focus on safety. The trial measures the incidence, nature, severity, and relationship of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and dose-limiting toxicities (DLTs) after DIAG723 is given. A TEAE is any unwanted medical event that appears or worsens after treatment starts. A serious adverse event is one that results in hospitalisation, is life-threatening, or causes lasting problems. A dose-limiting toxicity is a side effect severe enough to prevent increasing the dose. The trial also measures changes in laboratory parameters, vital signs, physical examination findings, and electrocardiogram (ECG) parameters, which record the heart's electrical activity.
Who can take part?
The trial enrols adults with hereditary hemorrhagic telangiectasia (HHT). The registry record does not provide further details on inclusion or exclusion criteria such as age limits beyond adulthood, specific HHT features, or previous treatments.
What side effects are known?
No side effects of DIAG723 are reported in the available sources. The trial is designed to collect information on treatment-emergent adverse events, serious adverse events, and dose-limiting toxicities, but results have not yet been published. Side effects will only be known once the study reports its findings.